The Breakthrough Therapy Designation (BTD) was granted based on the highly compelling results from the Phase 2 EMBOLD trial in SCN2A and SCN8A developmental and epileptic encephalopathies (DEEs) The EMBOLD cohort 2 pivotal trial is on track for topline results in H1 2026 with NDA filing to follow Praxis has recently initiated the EMERALD study investigating relutrigine broadly in DEEs BOSTON, July 17, 2025 (GLOBE NEWSWIRE) --...Read more
Breakthrough Therapy designation based on interim clinical data from Phase I/II trials showing clinically meaningful improvements in cardiac biomarkers and functional measures LX2006 also selected for FDA Chemistry, Manufacturing, and Controls Development and Readiness Pilot (CDRP) program, created to facilitate CMC registrational readiness and support faster patient access NEW YORK, July 07, 2025 (GLOBE NEWSWIRE) -- Lexeo...Read more
Phase 3 Aspire study enrollment on track to complete in 2025 Aurora study to evaluate GTX-102 in other Angelman syndrome genotypes and ages expected to initiate later this year NOVATO, Calif., June 27, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), today announced that it has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for GTX-102 (apazunersen) as a treatment...Read more
Breakthrough Therapy Designation based on promising early clinical evidence observed with daraxonrasib in patients with pancreatic ductal adenocarcinoma (PDAC) RASolute 302, a Phase 3 registrational study of daraxonrasib in patients with previously treated metastatic PDAC, expected to substantially complete enrollment this year REDWOOD CITY, Calif., June 23, 2025 (GLOBE NEWSWIRE) -- Revolution Medicines, Inc. (Nasdaq: RVMD), a...Read more
Based on Type C meeting and new data, Dyne submitted revised ACHIEVE trial protocol to FDA elevating vHOT to primary endpoint for U.S. Accelerated Approval New positive clinical data from Phase 1/2 ACHIEVE trial support vHOT as early indicator of clinical benefit with DYNE-101 in DM1 Ongoing Registrational Expansion Cohort in ACHIEVE trial to enroll 60 participants and include sites in U.S. Company to host an...Read more
Designation Supported by CLOVER WaM Phase 2 Study Data Which Reported an 83.6% Overall Response Rate (ORR) Seeking Guidance from EMA to Determine if CLOVER WaM Phase 2 Data Meets Criteria to Apply for Fast-Track, Conditional Marketing Authorization, Answer Expected Late July FLORHAM PARK, N.J., June 04, 2025 (GLOBE NEWSWIRE) -- Cellectar Biosciences, Inc. (NASDAQ: CLRB), a late-stage clinical biopharmaceutical company focused on...Read more
Designation is based on the pivotal Phase 3 DRAGON trial interim analysis results demonstrating Tinlarebant’s efficacy and favorable safety profile Trial completion expected by Q4 2025 (including a three-month follow-up period) Tinlarebant has previously been granted Fast Track and Rare Pediatric Disease Designations in the U.S., Orphan Drug Designation in the U.S., Europe, and Japan, and the Pioneer Drug Designation in Japan for...Read more
Breakthrough Therapy Designation request based on veligrotug’s (i) consistent and robust improvement and resolution of diplopia in chronic TED, and (ii) rapid onset of proptosis response Veligrotug met all of its primary and secondary endpoints in the pivotal THRIVE and THRIVE-2 clinical trials in active and chronic TED First and only drug candidate in chronic TED to demonstrate statistically significant and clinically...Read more
Registrational study in patients with tumor hyperinsulinism (HI) expected to commence mid-year Designation underscores need for therapies to treat severe hypoglycemia in the oncology setting REDWOOD CITY, Calif., May 05, 2025 (GLOBE NEWSWIRE) -- Rezolute, Inc. (Nasdaq: RZLT) (“Rezolute” or the “Company”), a late-stage rare disease company focused on treating hypoglycemia caused by HI, today announced that the U.S. Food and Drug...Read more
Breakthrough Therapy designation based on clinical evidence from Phase I/II trials showing meaningful slowing of disease progression Additional regulatory update and guidance on the Biologics License Application submission expected in the second quarter of 2025 LEXINGTON, Mass. and AMSTERDAM, April 17, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative...Read more
Designation enables expedited development and priority regulatory review BTD application was supported by updated clinical data from Ph2 neoadjuvant UM trial that we are targeting to present at medical conferences in mid-2025 and H2 2025 Targeting to initiate a Ph3 registrational study in neoadjuvant UM in H1 2025 Neoadjuvant UM has a projected annual incidence of ~12k patients, and is a high unmet medical with no FDA approved...Read more
UTRECHT, The Netherlands and CAMBRIDGE, Mass., Feb. 18, 2025 (GLOBE NEWSWIRE) -- Merus N.V. (Nasdaq: MRUS), a clinical-stage oncology company developing innovative, full-length multispecific antibodies and antibody drug conjugates (Biclonics®, Triclonics® and ADClonics®), for cancer, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation (BTD) to petosemtamab in combination with...Read more
Designation is based on TELLOMAK Phase 2 results demonstrating efficacy and a favorable safety profile in patients with advanced Sézary syndrome heavily pre-treated, post- mogamulizumab. Breakthrough Therapy Designation is intended to accelerate the development and regulatory review in the U.S. of drugs that are intended to treat a serious condition; adding to a Fast Track designation by the U.S. FDA received in 2019 as well as a PRIME...Read more
The Company will present at the Annual J.P. Morgan Healthcare Conference, providing business updates on its sarcoma franchise and other cell therapy pipeline assets Adaptimmune Allo-T program to be featured at the Biotech ShowCase(TM) and the Wuxi Global Forum 2025 Philadelphia, Pennsylvania and Oxford, United Kingdom--(Newsfile Corp. - January 13, 2025) - Adaptimmune Therapeutics plc (NASDAQ: ADAP), a company working to redefine...Read more
SOUTH SAN FRANCISCO, Calif., Jan. 08, 2025 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (NASDAQ: DNLI), today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for tividenofusp alfa (DNL310) for the treatment of individuals with Hunter syndrome (MPS II). This designation is in addition to Fast Track Designation, Orphan Drug Designation, and Rare Pediatric Disease Designation, all...Read more
Breakthrough Therapy Designation granted based on key positive data from the Phase 2b (RIZE) study and current unmet medical need in congenital hyperinsulinism (HI) Ersodetug continues to advance in clinical development as a potential treatment for hypoglycemia caused by all forms of hyperinsulinism; topline sunRIZE data expected second half of this year REDWOOD CITY, Calif., Jan. 07, 2025 (GLOBE NEWSWIRE) -- Rezolute, Inc. (Nasdaq:...Read more
FOSTER CITY, Calif. / Dec 17, 2024 / Business Wire / Gilead Sciences, Inc. (Nasdaq: GILD) today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to Trodelvy® (sacituzumab govitecan-hziy) for the treatment of adult patients with extensive-stage small cell lung cancer (ES-SCLC) whose disease has progressed on or after platinum-based chemotherapy. The Breakthrough Therapy Designation is...Read more
Designations aim to expedite the development and review of promising therapies for serious conditions with unmet medical needs Phase 3 ECLIPSE registrational program in chronic hepatitis delta to begin in the first half of 2025 SAN FRANCISCO / Dec 12, 2024 / Business Wire / Vir Biotechnology, Inc. (Nasdaq: VIR) today announced that tobevibart and elebsiran have received U.S. Food and Drug Administration (FDA) Breakthrough...Read more
First Breakthrough Therapy Designation for AstraZeneca and Daiichi Sankyo’s datopotamab deruxtecan Based on TROPION-Lung05 Phase II trial and supported by data from TROPION-Lung01 Phase III trial WILMINGTON, Del. / Dec 09, 2024 / Business Wire / Datopotamab deruxtecan (Dato-DXd) has been granted Breakthrough Therapy Designation (BTD) in the US for the treatment of adult patients with locally advanced or metastatic epidermal growth...Read more
Designation based on encouraging Phase 1b clinical data, including that SER-155 resulted in a 77% relative risk reduction in bacterial bloodstream infections versus placebo Breakthrough Therapy meeting with FDA on next study of SER-155 in allo-HSCT expected in Q1 2025 Seres seeking SER-155 strategic partnership to accelerate next study in allo-HSCT and expand to multiple target populations CAMBRIDGE, Mass., Dec. 09, 2024 (GLOBE...Read more
Supported by evidence from clinical studies that indicate that zorevunersen may demonstrate substantial improvement over available therapies Update on the company’s plans for a global, randomized, controlled Phase 3 registrational study anticipated by year-end BEDFORD, Mass. / Dec 04, 2024 / Business Wire / Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to restoring protein expression by...Read more
First breakthrough therapy designation for investigational sac-TMT in the U.S. RAHWAY, N.J. / Dec 03, 2024 / Business Wire / Merck (NYSE: MRK), known as MSD outside of the United States and Canada, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to sacituzumab tirumotecan (sac-TMT) for the treatment of patients with advanced or metastatic nonsquamous non-small cell lung...Read more
WOBURN, Mass., Nov. 21, 2024 (GLOBE NEWSWIRE) -- Replimune Group, Inc. (NASDAQ: REPL), a clinical stage biotechnology company pioneering the development of novel oncolytic immunotherapies, today announced that it has submitted a biologics license application (BLA) to the FDA for RP1 (vusolimogene oderparepvec) in combination with nivolumab for the treatment of adult patients with advanced melanoma who have previously received an anti-PD1...Read more
VAX-31 Infant Indication: Investigational New Drug Application Cleared by FDA; Company Expects to Initiate VAX-31 Infant Phase 2 Study by the End of January 2025 VAX-31 Adult Indication: Breakthrough Therapy Designation Granted by FDA; Company Plans to Initiate Adult Phase 3 Pivotal, Non-Inferiority Study by Mid-2025 VAX-31, Designed to Cover Currently Circulating and Historically Prevalent Strains, is Being Studied for the...Read more
The Breakthrough Therapy Designation (BTD) for investigational nipocalimab in Sjögren's disease, a prevalent autoantibody disease with no approved advanced therapies, is supported by results from the Phase 2 DAHLIAS study A greater than 70 percent relative improvement in systemic disease activity at Week 24 was demonstrated in study participants on average who received nipocalimab 15 mg/kg compared to participants who received...Read more
Designation based on positive interim analysis of Phase 2b VANTAGE study FOSTER CITY, Calif. / Oct 10, 2024 / Business Wire / Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to volixibat as a potential treatment for cholestatic pruritus in patients with primary biliary cholangitis (PBC). The regulatory designation is based on the...Read more
Designation is based on data from the clinical development program which demonstrated clinical proof of concept Felzartamab, an investigational anti-CD38 monoclonal antibody, is a potential first-in-class therapeutic candidate for a range of rare immune-mediated indications with planning underway for Phase 3 development FDA Breakthrough Therapy Designation is intended to expedite the development and review of drugs for serious or...Read more
NOVATO, Calif., Oct. 07, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that it has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for setrusumab (UX143) as a treatment to reduce the risk of fracture associated with osteogenesis imperfecta (OI) Type...Read more
Supported by positive data from Phase 2b FASCINATE-2 trial of denifanstat in patients with MASH Preparations are ongoing to initiate Phase 3 program for denifanstat by the end of 2024 SAN MATEO, Calif., Oct. 01, 2024 (GLOBE NEWSWIRE) -- Sagimet Biosciences Inc. (Sagimet, Nasdaq: SGMT), a clinical-stage biopharmaceutical company developing novel therapeutics targeting dysfunctional metabolic and fibrotic pathways, today...Read more
U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation and Rare Pediatric Disease designation for bexicaserin for the treatment of Dravet syndrome LA JOLLA, Calif. / Sep 19, 2024 / Business Wire / Longboard Pharmaceuticals, Inc. (Nasdaq: LBPH), a clinical-stage biopharmaceutical company focused on developing novel, transformative medicines for neurological diseases, today announced that the FDA has granted Rare...Read more
Breakthrough Therapy Designation was granted based on preliminary clinical evidence from the PROPEL 2 clinical trial, meeting the FDA’s requirement of potentially demonstrating substantial improvement in efficacy over available therapies on clinically significant endpoint(s) BridgeBio will leverage the benefits of Breakthrough Therapy Designation to expedite the development and regulatory review of infigratinib in the United States and...Read more
PALISADE Phase 3 results demonstrate plozasiran reduced triglycerides by 80% from baseline and reduced the risk of developing acute pancreatitis by 83% in patients with genetically confirmed and clinically diagnosed FCS1 PASADENA, Calif. / Sep 10, 2024 / Business Wire / Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that the United States Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to...Read more
SEATTLE and VANCOUVER, British Columbia, July 31, 2024 (GLOBE NEWSWIRE) -- Achieve Life Sciences, Inc. (Nasdaq: ACHV), a late-stage pharmaceutical company committed to the global development and commercialization of cytisinicline for smoking cessation and nicotine dependence, announced today that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for cytisinicline for nicotine e-cigarette, or vaping,...Read more
Astria Therapeutics is a biopharmaceutical company, and our mission is to bring life-changing therapies to patients and families affected by rare and niche allergic and immunological diseases. Our lead program, STAR-0215, is a monoclonal antibody inhibitor of plasma kallikrein in clinical development...
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